Jump to main content

Gen Therapie Study Hämophilia B

Final Analysis of Pivotal HOPE-B Study Demonstrates Durable and Sustained Therapeutic Effect of Etranacogene Dezaparvovec Gene Therapy in Hemophilia B – Data Presented at EAHAD 2022

  • Data from the largest gene therapy study in Hemophilia B to date shows that etranacogene dezaparvovec is statistically superior in reducing annualized bleeding rate compared to baseline FIX prophylactic therapy
  • Following a single administration of etranacogene dezaparvovec, participants maintained stable Factor IX (FIX) activity through 18 months 
  • Therapeutic benefit observed in patients with pre-existing anti-capsid neutralizing antibody levels up to a titer of 1:678